Evaluation of the efficacy of immunotherapy for non-resectable mucosal melanoma

Antoine Moya-Plana, Ruth Gabriela Herrera Gómez, Caroline Rossoni, Laurent Dercle, Samy Ammari, Isabelle Girault, Séverine Roy, Jean Yves Scoazec, Stephan Vagner, François Janot, Alexander M.M. Eggermont, Caroline Robert

Research output: Contribution to journalArticlepeer-review

46 Citations (Scopus)


Background: Immune checkpoint inhibitors are now standard-of-care treatments for metastatic cutaneous melanoma. However, for rare sub-groups, such as mucosal melanomas, few published data are available, and with no established therapeutic guidelines. Our objective was to assess the response to anti-CTLA4 and anti-PD1 immunotherapy in patients with mucosal melanomas. Methods: We performed a single-center, prospective cohort analysis of patients with non-surgical locally advanced and/or metastatic mucosal melanoma receiving anti-CTLA4 and/or anti-PD1 immunotherapy from 2010 to 2016. Results: Forty-four patients were enrolled, including 18 (40.9%) with head and neck, 12 (27.3%) with vulvo-vaginal and 14 (31.8%) with ano-rectal primary tumours. Eleven (25%) patients had stage 3 disease, and 11 (25%) had distant metastases. The first-line immunotherapy was ipilimumab in 24 patients and pembrolizumab in 20. The objective response rate (ORR) was 8.2% (one complete response) for ipilimumab and 35% (four complete responses) for pembrolizumab. No significant difference was observed for primary tumour location. The median follow-up was 24 months (range 4–73). The median progression-free survival (PFS) in the first-line ipilimumab and pembrolizumab groups was 3 months [95% confidence interval (CI) 2.5–4.6] and 5 months (95% CI 2.6–33.1), respectively (p = 0.0147). Conclusion: In the patients with unresectable and/or metastatic mucosal melanoma, we found ORR and PFS rates comparable to those in patients with cutaneous melanoma, with no significant differences in the types of mucosal surfaces involved. Anti-PD1 therapy has a more favorable benefit-risk ratio than ipilimumab and should be used preferentially.

Original languageEnglish
Pages (from-to)1171-1178
Number of pages8
JournalCancer Immunology, Immunotherapy
Issue number7
Publication statusPublished - 1 Jul 2019
Externally publishedYes


  • Anti-CTLA4
  • Anti-PD1
  • Immunotherapy
  • Ipilimumab
  • Mucosal melanoma
  • Pembrolizumab


Dive into the research topics of 'Evaluation of the efficacy of immunotherapy for non-resectable mucosal melanoma'. Together they form a unique fingerprint.

Cite this